Optimizing Early Ophthalmology Clinical Trials: Home OCT and Modeling Can Reduce Sample Size by 20% to 40.

ABSTRACT:

Purpose

Early-stage clinical trials for major retinal diseases face challenges due to substantial interpatient variability, end points with high intrapatient variability, and prolonged follow-up periods required to detect treatment effects. This study explores whether integrating home optical coherence tomography (OCT) monitoring with pharmacokinetic/pharmacodynamic (PK/PD) modeling can reduce clinical trial sample size.

Methods

A population PK/PD model was developed using longitudinal central subfield thickness data from a home OCT study. Monte Carlo simulations and bootstrapping were used to evaluate the impact of different monitoring strategies on sample size requirements to detect a simulated effect size of approximately 50-µm central subfield thickness reduction over an

SUBMITTER: Hermes J 

PROVIDER: S-EPMC12410251 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature